Patient Participation and the Power of Persuasion: A Qualitative Analysis of Patient Testimonies During FDA Drug Advisory Committees

CONTEXT: The US Food and Drug Administration invites public input at advisory committee meetings on drug approvals. We aimed to characterize how patients and caregivers who speak during these meetings establish legitimacy, present experiential knowledge as evidence, and seek to influence FDA decision-making. METHODS: We identified all FDA human drug advisory committee meetings from 2015 to 2023 that included at least one public speaker who self-identified as a patient or family member. Using publicly available transcripts, we conducted a thematic analysis of 844 testimonials from 155 meetings. FINDINGS: We found that testimonials consistently followed a four-part arc: First, speakers claimed authority through biographical detail and representative status, often combining professional expertise with personal illness to establish legitimacy and humanize their experiences. Second, they documented the burden of their disease, describing loss of function, family strain, financial consequences, and long histories of therapeutic failure or neglect. Third, they framed treatment experiences as real-world evidence, emphasizing meaningful gains in function, serious harms, and equity claims related to access and stigma. Finally, speakers addressed the FDA directly, casting officials as moral agents and asserting patient autonomy. CONCLUSIONS: Recognizing this framework can help clarify and inform guidance about how patient and caregiver testimonials should be incorporated into advisory committee and FDA drug approval decisions.